FDA approves hemophilia A drug for children
FDA recently expanded the use of antihemophilic factor (recombinant), pegylated (Adynovate, Shire plc) for hemophilia A patients under 12 years old.
FDA also approved the medicine, first approved in November, 2015, for use in surgical setting sfor both adult and pediatric patients. Adynovate, an extended circulating half-life recombinant factor VIII treatment, is built on the full-length antiphemophilic factor (recombinant) molecule (Advate), a hemophilia A treatment with more than 13 years of real-world patient experience.
The wholesale acquisition cost for Adynovate is $1.98 per 1 IU dose. However, the actual price paid by patients will differ based on their insurance coverage, Jeff Schaffnit, head of US Hematology Franchise for Shire, told FormularyWatch.
“Adynovate is an option for patients who may not be achieving their treatment goals. The treatment leverages proprietary PEGylation technology designed to extend the amount of FVIII available for use in the body,” Schaffnit said.
“Physicians need additional treatment options for pediatric patients and those undergoing surgery with hemophilia A,” said lead investigator Eric Mullins, MD, research director at the Hemophilia Treatment Center, Cincinnati Children’s Hospital Medical Center. “As someone who has routinely seen the benefits of factor replacement in adult patients with hemophilia A, I am pleased to be able to provide Adynovate to more patients.”
The approval of Adynovate to treat children under 12 years old was based on data from a prospective, uncontrolled, open-label, multicenter phase 3 trial designed to assess the immunogenicity along with the safety and efficacy of Adynovate. The drug met its primary end point with no previously treated children having developed inhibitory antibodies to Adynovate. In addition, no treatment-related serious adverse events were reported.
In addition, 73% of children had zero joint bleeds while on prophylactic treatment with Adynovate and 38% experienced zero bleeds. The median overall annualized bleeding rate (ABR) among pediatric patients treated with Adynovate was 2.0, similar to the rates seen in the adult study.
Approval to use Adynovate in surgical settings for both adult and pediatric patients was based on interim results of an ongoing phase 3 study of perioperative control of hemostasis among 15 patients with severe hemophilia A undergoing surgical procedures.